PAH

Diagonal Therapeutics Launches with $128 Million in Financing to Pioneer a New Approach to Discovering and Developing Agonist Antibodies to Tackle the Underlying Causes of Severely Debilitating Diseases

Retrieved on: 
Wednesday, April 3, 2024

CAMBRIDGE, Mass., April 03, 2024 (GLOBE NEWSWIRE) -- Diagonal Therapeutics, a biotechnology company pioneering a new approach to discovering and developing agonist antibodies, launched today with $128 million in financing. The Series A was co-led by BVF Partners and Atlas Venture, with participation from Lightspeed Venture Partners, RA Capital Management, Frazier Life Sciences, Viking Global Investors, Velosity Capital, and Checkpoint Capital. Diagonal was co-founded by Chief Executive Officer, Alex Lugovskoy, Ph.D., and Atlas and previously seeded by Atlas, Lightspeed Venture Partners, and Velosity Capital. The financing will support further advancement of the company’s proprietary DIAGONAL platform and pipeline of novel therapeutics to value-creating milestones, including its lead program for the treatment of hereditary hemorrhagic telangiectasia (HHT), a severely debilitating bleeding disorder with limited therapeutic options, through clinical proof-of-concept. Diagonal's agonist antibody activates a receptor complex in the TGF-β superfamily genetically impaired in HHT patients. In preclinical models of HHT, Diagonal's agonist antibodies prevent and reverse the formation of pathological vascular malformations.

Key Points: 
  • Diagonal was co-founded by Alex Lugovskoy, Ph.D., and Atlas and previously seeded by Atlas, Lightspeed Venture Partners, and Velosity Capital.
  • CAMBRIDGE, Mass., April 03, 2024 (GLOBE NEWSWIRE) -- Diagonal Therapeutics, a biotechnology company pioneering a new approach to discovering and developing agonist antibodies, launched today with $128 million in financing.
  • Diagonal was co-founded by Chief Executive Officer, Alex Lugovskoy, Ph.D., and Atlas and previously seeded by Atlas, Lightspeed Venture Partners, and Velosity Capital.
  • In preclinical models of HHT, Diagonal's agonist antibodies prevent and reverse the formation of pathological vascular malformations.

Inhibikase Therapeutics Announces Pre-IND Meeting with the FDA for IkT-001Pro in Pulmonary Arterial Hypertension

Retrieved on: 
Wednesday, April 3, 2024

The meeting will be held on April 5, 2024, with meeting results to be reported following receipt of the formal meeting minutes.

Key Points: 
  • The meeting will be held on April 5, 2024, with meeting results to be reported following receipt of the formal meeting minutes.
  • “Following our pre-NDA discussion with the FDA related to the path to approval for IkT-001Pro in up to 11 blood and stomach cancers in January, we requested an additional FDA meeting with the Division of Cardiology and Nephrology to discuss Pro as a treatment for Pulmonary Arterial Hypertension,” said Dr. Milton Werner, President and Chief Executive Officer of Inhibikase.
  • “PAH is a rare condition that primarily afflicts women between the ages of 30 and 60 and can lead to premature heart failure and death.
  • We believe that Pro may be a be a safer and better tolerated therapeutic option for imatinib treatment in PAH.

Update on Favorable Legal and Regulatory Outcomes Clearing Path for Potential FDA approval of YUTREPIA™ (treprostinil) inhalation powder

Retrieved on: 
Monday, April 1, 2024

As a result, the U.S. Food and Drug Administration (FDA) is no longer enjoined from issuing final approval of Liquidia’s New Drug Application (NDA) for YUTREPIA™ (treprostinil) inhalation powder.

Key Points: 
  • As a result, the U.S. Food and Drug Administration (FDA) is no longer enjoined from issuing final approval of Liquidia’s New Drug Application (NDA) for YUTREPIA™ (treprostinil) inhalation powder.
  • We have submitted the judge’s order to the FDA and look forward to a decision from the FDA in the near future.
  • Our commercial team is fully prepared to launch YUTREPIA in both PAH and PH-ILD should the FDA grant final approval.
  • The FDA is now able to take final action on YUTREPIA’s amended NDA that seeks approval for both indications.

Orphan designation: imatinib Treatment of pulmonary arterial hypertension, 21/06/2021 Positive

Retrieved on: 
Tuesday, April 9, 2024

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of pulmonary arterial hypertension (PAH) in the European Union on 21 June 2021.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024

Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Key Points: 


Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Aerovate Therapeutics Announces Full-Year 2023 Financial Results and Business Highlights

Retrieved on: 
Monday, March 25, 2024

Cash, cash equivalents and short-term investments totaled $122.4 million as of December 31, 2023, compared to $135.2 million as of September 30, 2023.

Key Points: 
  • Cash, cash equivalents and short-term investments totaled $122.4 million as of December 31, 2023, compared to $135.2 million as of September 30, 2023.
  • The decrease was primarily driven by operational costs for the three-month period ended December 31, 2023.
  • Net loss: Net loss for the year ended December 31, 2023 was $75.5 million as compared to $51.5 million for the year ended December 31, 2022.
  • Net loss included stock-based compensation expense of $11.9 million and $5.5 million for the years ended December 31, 2023 and December 31, 2022, respectively.

Liquidia Corporation Reports Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Wednesday, March 13, 2024

ET

Key Points: 
  • ET
    MORRISVILLE, N.C., March 13, 2024 (GLOBE NEWSWIRE) -- Liquidia Corporation (NASDAQ: LQDA) (Liquidia or the Company) today reported financial results for the full year ended December 31, 2023.
  • ET to discuss the 2023 financial results and provide a corporate update.
  • In December 2023, the previous ruling by the Patent Trial and Appeal Board (PTAB) that all of the claims in U.S. Patent No.
  • The year ended December 31, 2023 included a $2.3 million loss on extinguishment of debt related to repayment of the A&R SVB LSA in January 2023.

FDA Approves Merck’s WINREVAIR™ (sotatercept-csrk), a First-in-Class Treatment for Adults with Pulmonary Arterial Hypertension (PAH, WHO* Group 1)

Retrieved on: 
Tuesday, March 26, 2024

“The Pulmonary Hypertension Association welcomes the development of new therapies for those with PAH,” said Matt Granato, president and chief executive officer, Pulmonary Hypertension Association.

Key Points: 
  • “The Pulmonary Hypertension Association welcomes the development of new therapies for those with PAH,” said Matt Granato, president and chief executive officer, Pulmonary Hypertension Association.
  • In the WINREVAIR treatment group, the placebo-adjusted median increase in 6MWD was 41 meters (95% CI: 28, 54; p
  • The median treatment difference in PVR between WINREVAIR and placebo was -235 dynes*sec/cm5 (95% CI: -288, -181; p
  • The median treatment difference in NT-proBNP between WINREVAIR and placebo was -442 pg/mL (95% CI: -574, -310; p

Gossamer Bio Announces Appointment of Steven D. Nathan, M.D., and Skye Drynan to its Board of Directors

Retrieved on: 
Tuesday, March 12, 2024

Skye Drynan is the Founder, CEO and Creative Director of House of Skye, Ltd., and a former Partner and Senior BioPharma Analyst at Capital Group.

Key Points: 
  • Skye Drynan is the Founder, CEO and Creative Director of House of Skye, Ltd., and a former Partner and Senior BioPharma Analyst at Capital Group.
  • Ms. Drynan joins the Board with an extensive background in finance, investment, and entrepreneurship, specializing in the biotechnology industry.
  • “The addition of Dr. Nathan to our Board signifies a pivotal moment in Gossamer’s evolution,” said Faheem Hasnain, Co-Founder, Chairman and CEO of Gossamer Bio.
  • Ms. Drynan is the Founder, CEO and Creative Director of House of Skye, Ltd., which she founded in 2016.

U.S. FDA Approves OPSYNVI® (macitentan and tadalafil) as the First and Only Once-Daily Single-Tablet Combination Therapy for Patients with Pulmonary Arterial Hypertension (PAH)

Retrieved on: 
Friday, March 22, 2024

RARITAN, N.J., March 22, 2024  /PRNewswire/ -- Johnson & Johnson today announced that the U.S. Food and Drug Administration (FDA) has approved OPSYNVI® – a single-tablet combination of macitentan, an endothelin receptor antagonist (ERA), and tadalafil, a phosphodiesterase 5 (PDE5) inhibitor – for the chronic treatment of adults with pulmonary arterial hypertension (PAH, World Health Organization [WHO] Group I) and WHO functional class (FC) II-III.1 OPSYNVI® may be used in patients with PAH who are treatment-naïve or who are already on an ERA, PDE5 inhibitor or both. OPSYNVI® may be used in patients who are currently treated concomitantly with stable doses of macitentan 10 mg and tadalafil 40 mg (20 mg x 2) as separate tablets.1

Key Points: 
  • Historically, this required patients to take multiple pills because no single-tablet combination therapy targeting two or more pathways was available," said Kelly Chin, M.D., Professor of Internal Medicine and Director of the Pulmonary Hypertension Program at UT Southwestern Medical Center, and an investigator in the A DUE study.
  • The FDA's approval of OPSYNVI® is based on the results from the pivotal Phase 3 A DUE study , in which OPSYNVI® demonstrated greater reduction in Pulmonary Vascular Resistance (PVR) after 16 weeks versus tadalafil or macitentan monotherapy.
  • "People with PAH often live with the burden of taking many pills each day, which can pose challenges," said James F. List, M.D., Ph.D., Global Therapeutic Area Head, whose team oversees a portfolio of programs including Pulmonary Hypertension at Johnson & Johnson.
  • "We're thrilled to bring this single-tablet combination therapy to patients, as it has the potential to optimize disease management and fulfill a significant unmet need in supporting recently updated treatment guidelines that call for initial or early combination treatment."